gMG patients can safely use self-injectors for experimental therapy
Simulated-use study shows participants were able to complete most injections
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- Generalized myasthenia gravis is an autoimmune condition characterized by muscle weakness and fatigue.
- Patients can safely and effectively self-administer the experimental therapy gefurulimab using prefilled syringes or autoinjectors.
- Clinical trial results support self-administration devices as a user-friendly treatment approach for managing symptoms.
A prefilled syringe and an autoinjector used to deliver gefurulimab, an experimental therapy in late-stage development for generalized myasthenia gravis (gMG), can be safely and effectively used by most gMG patients, caregivers, and healthcare professionals.
That’s according to a simulated-use study, which aims to assess if people can safely and effectively use a medical product, but where the actual drug is not delivered. Participants were able to complete most injections, with few use-related difficulties, data showed.
These findings support gefurulimab’s prefilled syringe and autoinjector “as convenient and user-friendly options for self-administration of medication by patients with gMG and their caregivers,” researchers wrote.
The study, “Usability and Acceptance of Autoinjector and Prefilled Syringe Devices by Patients with Generalized Myasthenia Gravis in a Human Factors Study,” was published in Advances in Therapy. It was funded by Alexion, Astrazeneca Rare Disease, gefurulimab’s developer, and some of its authors are company employees.
Gefurulimab outperformed placebo at reducing symptoms
gMG is an autoimmune disease marked by symptoms such as muscle weakness and fatigue. Most patients have self-reactive antibodies against acetylcholine receptors (AChRs), proteins involved in nerve-muscle communication. These antibodies can activate the complement pathway, a part of the immune system, which is thought to contribute to the damaging autoimmune response that drives gMG.
Gefurulimab is an experimental antibody-based therapy designed to suppress complement activation by blocking the complement protein C5. In a large, global Phase 3 clinical trial called PREVAIL (NCT05556096), the therapy was found to be superior to a placebo at reducing gMG activity and symptoms.
Because of its tiny size, gefurulimab can be administered under the skin (subcutaneously) using either a prefilled syringe or an autoinjector. Both devices allow treatment to be self-administered by patients or caregivers after appropriate training.
“Device usability by patients is important, as symptoms associated with gMG, such as muscle weakness and [double vision], may impair the ability of patients to self-administer injectable therapies,” the researchers wrote.
Participants included patients, caregivers, healthcare professionals
After several previous were studies conducted to assess the overall usability and comprehensibility of the design, labeling, instructions, and packaging of both gefurulimab injection devices, researchers at Alexion, VCU Health, and Insights by Nemera conducted a final validation simulated-use study.
The study was designed to assess whether intended users can safely and effectively use both gefurulimab injection devices and understand their labeling. Regulatory agencies such as the U.S. Food and Drug Administration require companies to conduct these tests to spot user mistakes before a therapy enters the market.
The final study involved 75 adult participants, including 30 gMG patients, 30 caregivers, and 15 healthcare professionals. It was conducted at nine sites in the U.S., including a market research facility, usability laboratories, and participants’ homes.
Patients and caregivers were assigned to simulate one or two injections using the prefilled syringe or autoinjector. Healthcare providers, who were mainly registered nurses, were assigned to simulate only the use of the autoinjector.
Overall, 94% of all simulated-use tasks with the prefilled syringe were completed successfully. Also, 90% of participants (27 of 30) “successfully determined the number of injections required to administer the prescribed dose … and successfully pushed the plunger all the way to administer the full dose into the injection pad,” the team wrote.
Three participants did not administrate the full dose because of issues with needle placement, removing the needle too early, or not recalling that two injections were required. These issues occurred only once each, with no recurring patterns identified.
No changes recommended to either device
The autoinjector showed similar usability, with 96% of tasks completed successfully and 87% of participants (39 of 45) — including all patients — successfully administering their assigned dose.
Overall, the researchers concluded that identified use-related risks were acceptable and did not recommend changes to either device.
Participants also completed 12 knowledge tasks to assess whether they could find and understand important information in the product labeling and instructions for use, including storage, dosing, and device-use procedures.
Overall, 97.2% of datapoints for knowledge tasks involving the prefilled syringe and 99% of those involving the autoinjector were answered correctly in simulated-use testing. The main difficulties involved recognizing whether the medication looked suitable for use, understanding where to give a second injection, and knowing what to do if the prefilled syringe carton was damaged.
After completing the simulated-use scenarios, patients rated their experience with the assigned device using a questionnaire covering ease of use, intuitiveness, comfort, confidence, convenience, and overall acceptability. Ratings ranged from 1 (low) to 5 (high).
Most patients (93%) who used the prefilled syringe and all of those using the autoinjector found their device acceptable. Positive ratings across the six attributes ranged from 87% to 93% for the prefilled syringe and from 93% to 100% for the autoinjector.
The study demonstrated that both devices can be used safely and effectively for “self-injection in the intended use settings by untrained patients, caregivers, and [healthcare professionals] without patterns of preventable use deviations that could result in harm to the user or patient,” the researchers concluded.
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